The Rare Disease Company Coalition (RDCC) has issued a new resource outlining a set of policy recommendations that would change the nature of rare disease diagnosis in the United Sates. The recommendations find basis in the fact that the earlier patients receive an accurate diagnosis, the better their chances are of accessing effective therapies.

In issuing this new resource, the RDCC calls on policymakers and regulators to better implement existing technology and proven public health programs to ensure the healthcare system keeps pace with innovation. In modernizing the system, these policy solutions will not only ensure patients can access new therapies when needed most, but also promote the more rapid adoption of new discoveries.

The new resource also includes details on how government entities can evolve to better support early diagnosis through improved collaboration and communication. Increasing coordination and streamlining practices throughout the entire healthcare ecosystem will better equip providers and specialists to make accurate diagnosis and care for rare disease patients.

Enabling Earlier Rare Disease Diagnosis

The Rare Disease Company coalition (RDCC) is pleased to share this new resource for federal and state policymakers and regulators to help inform legislative actions that can bolster patient access to life-changing, FDA-approved rare disease therapies.

Today, over 95% of rare diseases lack an FDA-approved treatment, and while the FDA continues to add to the number approved therapies, treatments are only as valuable as they are accessible.

Once a rare disease treatment is approved by the FDA, patients seeking to benefit from the approved therapy are often faced with challenges to access due to complex interstate credentialing, limited coverage, delays in review, inaccurate value frameworks, and insufficient medical necessity determinations.

In this new resource, the RDCC outlines six areas for improvement with specific policy solutions that will improve outcomes for the rare disease community.

2023 Access Campaign Report by Rare Disease Company Coalition
Advancing Patient Access to Rare Disease Therapies